On July 20, 2022, Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter) reported that its CSF-1R inhibitor ABSK021 had been granted the breakthrough therapy designation from Center for Drug Evaluation, NMPA for the treatment of tenosynovial giant cell tumor (TGCT) that not amenable to surgery (Press release, Abbisko Therapeutics, JUL 20, 2022, View Source [SID1234633494]).

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The Breakthrough Therapy Designation is a CDE program for innovations during clinical trials that are used for the prevention and treatment of serious life-threatening diseases or seriously affect the quality of life, and there are no effective prevention and treatment methods or there is sufficient evidence to show that they have obvious clinical advantages compared with existing treatment methods.

This breakthrough therapy designation approval is based on results from the phase Ib clinical trial of TGCT cohort in China for CSF-1R inhibitor ABSK021.

TGCT, also known as pigmented villonodular synovitis, is a locally aggressive neoplasm that affects synovial joints, mucous sacs, and tendon membranes, resulting in swelling, pain, stiffness, and decreased activity of the affected joints which seriously affect the patient’s quality of life.

According to the 2013 World Health Organization classification, TGCTs were classified as localized TGCT and diffuse TGCT. Compared with localized TGCT (80%-90%), the incidence rate of diffuse TGCT is lower (10-20%).

Overexpression of colony-stimulating factor 1(CSF1) occurs in most TGCTs. Surgical resection is the standard treatment for TGCT. However, not all patients are suitable for surgical treatment. It is difficult to remove tumors of diffuse patients by surgery, which may possibly lead to severe joint damage, total synovectomy, joint replacement, or even amputation, and the risk of surgical complications can be high. It has been reported that more than 50% of patients with diffuse TGCT will undergo recurrence after surgical resection. For those TGCT patients not amenable to surgery, there is currently no approved drug available in China.

ABSK021 is a novel, orally available, highly selective, and highly potent small molecule inhibitor of CSF-1R, independently developed by Abbisko Therapeutics. It is also the first selective CSF-1R inhibitor discovered by a Chinese company that has advanced into human clinical trial. A number of studies have shown that blocking the CSF-1R signaling pathway could effectively modulate and change macrophage functions, and potentially treat many macrophage-dependent human diseases. Abbisko has completed a phase Ia dose escalation study for ABSK021 in the U.S., with phase Ib expansion ongoing in both U.S. and China. In addition to TGCT, Abbisko is actively exploring the potential of ABSK021 in treating other indications including many types of solid tumors in clinic, and has collaborated with Sperogenix in exploring its potential for treating ALS and other CNS diseases. As of the date of this press release, no high-selective CSF-1R inhibitor has been approved in China.

On July 20, 2022 Manhattan BioSolutions, Inc. reported it has entered into two commercial evaluation license agreements with the National Cancer Institute (NCI), part of the US National Institutes of Health (NIH), to generate and validate novel antibody-drug conjugate (ADC) therapies using monoclonal antibodies developed by NCI (Press release, Manhattan BioSolutions, JUL 20, 2022, View Source [SID1234618289]). ADCs harness the targeting ability of antibodies to deliver drugs to the tumor microenvironment or directly to cancer cells.

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Under the first agreement, Manhattan BioSolutions obtained the proprietary monoclonal antibody discovered and validated by Drs. Ira Pastan, MD, Distinguished Investigator, Chief Emeritus of the Laboratory of Molecular Biology and Mitchell Ho, PhD, Director of the Antibody Engineering Program and Deputy Chief of the Laboratory of Molecular Biology at the NCI Center for Cancer Research. This antibody targets a cell surface glycoprotein overexpressed in multiple cancers including mesothelioma, lung, ovarian and pancreatic cancer. Dr. Ira Pastan is recognized for his pioneering contributions to the fields of receptor biology, which led to the discovery of the new class of antibody drugs called recombinant immunotoxins. The targeted immunotoxin he developed to treat a deadly form of leukemia is now an approved medication marketed by Astra Zeneca under the name Lumoxiti.

The second license agreement provides access rights toa new monoclonal antibody directed to a cell surface receptor tyrosine kinase overexpressed and mutationally activated in a rare pediatric cancer as well as in other solid tumors. This antibody was discovered by the team of Dr. Javed Khan, MD, Senior Investigator and Deputy Chief at the Genetics Branch at the NCI Center for Cancer Research.

The partnerships allow Manhattan BioSolutions to further expand its preclinical pipeline of innovative biologic agents for oncology applications. The antibodies discovered at NCI will be combined with the proprietary immune-stimulating linker-payloads invented by Dr. L. Nathan Tumey, an ex-Pfizer medicinal chemist, who is currently an Associate Professor in the School of Pharmacy and Pharmaceutical Sciences at Binghamton University. The current collaboration follows the previously announced grant award and collaborative research agreement with the laboratory of Dr. Dhaval Shah, a former Pfizer scientist, now an Associate Professor at the Department of Pharmaceutical Sciences, University at Buffalo.

"The synergistic combination of assets and platforms enables the discovery of novel classes of medicines designed to activate innate, microbial host-defense immunity in tumor microenvironment," said Dr. Borys Shor, CEO of Manhattan BioSolutions. "We have assembled a world-class team of drug discovery experts to develop next-generation ADCs: a trio of ex-Pfizer scientific leaders, including Drs. Dhaval Shah and Nathan Tumey, all with a proven track record of bringing transformative oncology medicines to clinical development."

On July 20, 2022 Singapore-based biotech start-up, Tetris Therapeutics Pte. Ltd. reported that it has changed its name to Axcynsis Therapeutics Pte. Ltd (Press release, Axcynsis Therapeutics, JUL 20, 2022, View Source [SID1234618243]).. The new name will better reflect the company’s core business and platform technology – AXC (Antibody-X Conjugate) Synthesis.

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"Antibody Drug Conjugates (ADCs) are a class of anticancer agents designed by conjugating cytotoxic drugs to monoclonal antibodies." said Dr. Zou Bin, founder and CEO of Axcynsis Therapeutics. "In fact, this concept can be extended beyond cytotoxic drugs. Hence we are developing our platform technologies to synthesize Antibody-X Conjugates, where "X" could be any molecules that disrupt the abnormal cells."

Axcynsis Therapeutics will continue to strengthen the company’s pipeline and provide new treatment options to address unmet medical needs.

On July 20, 2022 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Verve Therapeutics, Inc. (Nasdaq: VERV) reported an exclusive, four-year global research collaboration focused on discovering and developing an in vivo gene editing program for a single undisclosed liver disease (Press release, Vertex Pharmaceuticals, JUL 20, 2022, View Source [SID1234616853]).

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Under the terms of the collaboration, Verve will advance the discovery, research and certain preclinical development of a novel in vivo gene editing program for the target of interest, with all program costs funded by Vertex. Vertex will be responsible for subsequent development, manufacturing and commercialization of any program stemming from Verve’s research efforts.

"This partnership with Vertex enables an important step forward for Verve as we build out our leading gene editing capabilities and pipeline of in vivo gene editing medicines to address serious diseases," said Sekar Kathiresan, M.D., Co-Founder and Chief Executive Officer of Verve. "This agreement validates the pioneering work at Verve to develop liver-directed gene editing medicines and expands the reach of our capabilities and breadth of our pipeline. We are thrilled to partner with Vertex to advance this important research and development program."

"Vertex is committed to discovering and developing transformative medicines for people with serious diseases," said David Altshuler, M.D., Ph.D., Executive Vice President, Global Research and Chief Scientific Officer of Vertex. "We are impressed by the progress Verve has made and look forward to combining the expertise in gene editing and drug development of our two companies to serve more patients in need."

Transaction Terms
Under the terms of the agreement, Verve will receive an upfront payment of $60 million, including a $35 million equity investment in Verve. Verve is also eligible to receive up to $66 million in success payments, up to $340 million in development and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement.

On July 20, 2022 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it will host a webcast and conference call on August 4, 2022, at 4:30 p.m. ET to discuss its financial results for the fiscal 2022 third quarter ended June 30, 2022 (Press release, Arrowhead Research Corporation, JUL 20, 2022, View Source [SID1234616852]).

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 888-243-4451 or 412-542-4135 and ask to join the Arrowhead Pharmaceuticals call.

A replay of the webcast will be available on the Company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 877-344-7529 or 412-317-0088 and provide Conference ID 8080876.